PMID- 34889371 OWN - NLM STAT- MEDLINE DCOM- 20211221 LR - 20220327 IS - 1520-4383 (Electronic) IS - 1520-4391 (Print) IS - 1520-4383 (Linking) VI - 2021 IP - 1 DP - 2021 Dec 10 TI - Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks? PG - 190-195 LID - 10.1182/hematology.2021000322 [doi] AB - In case 1, a 14-month-old male child with sickle cell disease (SCD) was referred for evaluation for an allogeneic hematopoietic stem cell transplant (HCT). The patient had a history of dactylitis 3 times in his first year of life and febrile episodes twice at the consult. His 4-year-old sister was found to be human leukocyte antigen (HLA) identical. The patient was started on hydroxyurea (HU) at 2.5 years of age. His parents again sought consultation when he was 5 years old because of concerns about his medical condition. At the time, the patient had experienced 2 vaso-occlusive pain episodes (VOEs) requiring hospitalization during the previous 2 years. He had also experienced intermittent pain crises requiring rest at home for 2 to 3 days. The child has not attended school in person due to the COVID-19 pandemic. The family is considering HCT but is ambivalent about it because of potential toxicity. In case 2, an 8-year-old female child is 3 years out from HCT for SCD from her HLA-identical sibling. Before HCT, despite receiving HU, she had experienced >5 VOEs requiring hospitalization and 2 episodes of acute chest syndromes in the previous 3 years. She had also been missing almost 50 days of school days each year. After HCT, she is now attending school regularly and participating in all normal age-appropriate activities. The parents believe that HCT has been transformative in their child's life. CI - Copyright (c) 2021 by The American Society of Hematology. FAU - Shah, Niketa AU - Shah N AD - Section of Pediatric Hematology/Oncology/BMT, Yale School of Medicine, New Haven, CT. FAU - Krishnamurti, Lakshmanan AU - Krishnamurti L AD - Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA. LA - eng GR - UL1 TR001863/TR/NCATS NIH HHS/United States PT - Case Reports PT - Journal Article PT - Review PL - United States TA - Hematology Am Soc Hematol Educ Program JT - Hematology. American Society of Hematology. Education Program JID - 100890099 RN - 0 (HLA Antigens) SB - IM MH - Age Factors MH - Anemia, Sickle Cell/epidemiology/*therapy MH - Child MH - Child, Preschool MH - Evidence-Based Medicine MH - Female MH - HLA Antigens/analysis MH - *Hematopoietic Stem Cell Transplantation/adverse effects/methods MH - Humans MH - Infant MH - Male MH - Quality of Life MH - Risk Factors MH - Siblings MH - Tissue Donors PMC - PMC8791135 COIS- Niketa Shah: no competing financial interests to declare. Lakshmanan Krishnamurti: no competing financial interests to declare. EDAT- 2021/12/11 06:00 MHDA- 2021/12/22 06:00 PMCR- 2021/12/10 CRDT- 2021/12/10 08:45 PHST- 2021/12/10 08:45 [entrez] PHST- 2021/12/11 06:00 [pubmed] PHST- 2021/12/22 06:00 [medline] PHST- 2021/12/10 00:00 [pmc-release] AID - 482945 [pii] AID - 2021000322 [pii] AID - 10.1182/hematology.2021000322 [doi] PST - ppublish SO - Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):190-195. doi: 10.1182/hematology.2021000322.